Abstract: The present invention refers to mesenchymal stem cells (MSCs) characterized in that they are transduced with an integrative expression vector in order to stably co-express the chemokine receptor type 4 CXCR4 and the interleukin EL-10. The present invention also refers to the use of said MSCs as a medicament, particularly in the treatment of inflammatory and/or autoimmune diseases.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.

Abstract: The present invention relates generally to methods for genetic modification of hematopoietic cells. In particular, the invention relates to use of Prostaglandin E2 (PGE2), poloxamer, and protamine sulfate to enhance transduction by a recombinant retroviral vector. The compositions and methods of the present disclosure are particularly suitable for gene therapy applications, including the treatment of monogenic genetic diseases and disorders.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.

Abstract: The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.

Abstract: The present invention relates to the medical field, in particular to gene editing as a therapeutic approach for the treatment of metabolic diseases affecting the erythroid lineage in a mammalian subject. In invention particular embodiment it refers to the combination of cell reprograming and gene editing for PKD correction as a first example of the potential application of these advanced technologies to metabolic diseases affecting the erythroid lineage. In this sense, PKD patient-specific iPSCs were efficiently generated from PB-MNCs (peripheral blood mononuclear cells) by a SeV non-integrative system and efficiently use to treat pyruvate kinase deficiency. The gene editing strategy for PKLR gene correction was also successfully applied directly to hematopoietic progenitors.

Abstract: Methods and compositions for the generation and use of genetically corrected induced pluripotent stem cells are provided.

Abstract: The present invention relates to the use of the HS1 promoter of the vav oncogene in the production of vectors selected from the groups of integrative vectors and non-integrative, non-plasmid vectors, for use in the preparation of pharmaceutical compositions intended for somatic gene therapy. By generating vectors that contain the HS1 promoter of the vav oncogene, it was possible to generate vectors in which the marker transgene or therapeutic transgene is expressed at moderate, but stable, levels in various cell lines both in vitro and in vivo.